PEDIATRIC MEDICINES: GLOBAL MARKETS

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1 PEDIATRIC MEDICINES: GLOBAL MARKETS PHM170A October 2014 Dr. Cheryl Lee Barton Project Analyst ISBN: BCC Research 49 Walnut Park, Building 2 Wellesley, MA USA (toll-free within the USA), or (+1)

2 TABLE OF CONTENTS CHAPTER 1 INTRODUCTION 2 STUDY GOALS AND OBJECTIVES 2 REASONS FOR DOING THE STUDY 2 SCOPE OF REPORT 2 INTENDED AUDIENCE 2 METHODOLOGY AND INFORMATION SOURCES 3 ANALYSTS' CREDENTIALS 3 RELATED BCC RESEARCH REPORTS 4 BCC RESEARCH WEBSITE 4 DISCLAIMER 4 CHAPTER 2 SUMMARY 6 SUMMARY TABLE GLOBAL PEDIATRIC MARKET, 7 SUMMARY FIGURE GLOBAL PEDIATRIC MARKET, ($ MILLIONS) 7 CHAPTER 3 OVERVIEW 9 PEDIATRIC POPULATION 9 TABLE 1 GLOBAL PEDIATRIC MARKET BY REGION, 10 FIGURE 1 HISTORIC ANNUAL GROWTH RATES OF PEDIATRIC THERAPEUTIC SEGMENTS, (%) TABLE 2 DISTRIBUTION OF WORLD POPULATION IN THE 0-14-YEAR AND YEAR AGE GROUPS BY REGION, 2013 AND 2050 (%) TABLE 3 DISTRIBUTION OF WORLD POPULATION BY REGION IN THE 0-14-YEAR AGE GROUP, (%) TABLE 4 DISTRIBUTION OF WORLD POPULATION IN THE 0-14-YEAR AGE GROUP, (MILLIONS) OFF-LABEL DRUG USE 12 TABLE 5 OFF-LABEL CATEGORIES 13 TABLE 6 TYPES OF STUDIES REQUIRED WHEN MAKING A PIP SUBMISSION (NUMBER/%) AREAS OF UNMET CLINICAL NEED 16 TABLE 7 KEY AREAS OF UNMET PEDIATRIC NEED CITED BY THE EMA MARKET RESISTORS AND DRIVERS 18 TABLE 8 DIFFERENCES BETWEEN ADULT AND PEDIATRIC TRIAL DESIGN 19 TABLE 9 AIMS OF THE PEDIATRIC FORMULATIONS INITIATIVE 20 ADDRESSABLE POPULATION 21 TABLE 10 PERCENTAGE OF POPULATION BETWEEN 0 AND 14 YEARS OF AGE BY REGION, 2013 (%) GEOGRAPHIC AND THERAPEUTIC SEGMENTATION 23 TABLE 11 COMMON PEDIATRIC DISORDERS IN THE U.S. IN 2010 (MILLION/%) CHAPTER 4 REGULATORY LANDSCAPE 27 THE UNITED STATES 27 TABLE 12 COMPARISON OF THE MAIN FEATURES OF THE U.S. PEDIATRIC INCENTIVE PROGRAMS FIGURE 2 MAJOR MILESTONES OF PEDIATRIC LEGISLATION IN THE U.S THE EUROPEAN UNION 30 28

3 FIGURE 3 GENERAL PRINCIPLES GUIDING PEDIATRIC DRUG DEVELOPMENT - THE ICH E-11 GUIDELINE FIGURE 4 MAJOR MILESTONES OF PEDIATRIC LEGISLATION IN THE EU PEDIATRIC INVESTIGATION PLAN 32 TABLE 13 CLASSIFICATION OF PEDIATRIC AGE CATEGORIES IN THE EU AND THE U.S. PEDIATRIC COMMITTEE 33 REWARDS AND INCENTIVES 33 TABLE 14 PROCEDURAL GUIDELINES FOR PEDIATRIC DRUG DEVELOPMENT FOR EUROPE AND THE U.S. TABLE 15 SCIENTIFIC GUIDELINES FOR PEDIATRIC DRUG DEVELOPMENT FOR EUROPE AND THE U.S. JAPAN 35 AUSTRALIA 36 CANADA 38 TABLE 16 CURRENT LEGISLATIVE / REGULATORY PEDIATRIC INITIATIVES ACROSS KEY COUNTRIES GLOBALLY THE IMPACT OF NEW PEDIATRIC LEGISLATION 40 TABLE 17 KEY GLOBAL PEDIATRIC INITIATIVES CHAPTER 5 BARRIERS TO ENTRY 44 ACCESS TO SAFE, AFFORDABLE AND EFFECTIVE MEDICINES 44 TABLE 18 PRIORITY MEDICINES FOR CHILDREN UNDER FIVE YEARS OF AGE FOR MAJOR CAUSES OF MORTALITY, MORBIDITY, PALLIATIVE CARE AND CHILD SURVIVAL TABLE 19 PRIORITY MEDICINES FOR CHILD HEALTH AND SURVIVAL IN NEED OF FURTHER RESEARCH AND DEVELOPMENT PEDIATRIC CLINICAL TRIALS 47 FIGURE 5 PEDIATRIC AND ADULT TRIALS AS A PERCENTAGE, (%) 47 TABLE 20 TOP 10 COUNTRIES RANKED BY STUDY PARTICIPATION AND PATIENT ENROLLMENT FOR STUDIES ON DRUGS (NUMBER) TABLE 21 TOP 10 COUNTRIES RANKED BY STUDY PARTICIPATION AND PATIENT ENROLLMENT FOR STUDIES ON VACCINES (NUMBER) TRIAL DESIGNS 49 ETHICAL CONSIDERATIONS 49 RECRUITMENT AND RETENTION 50 AGE-APPROPRIATE FORMULATIONS 50 PRODUCT APPROVAL 51 UNITED STATES 51 EUROPEAN UNION 53 JAPAN 54 PRICING REIMBURSEMENT 55 UNITED STATES 55 EUROPEAN UNION 57 JAPAN CHAPTER 6 ADVANCES IN PEDIATRIC MEDICINES 60 THERAPEUTIC AREAS FOR PEDIATRIC DRUG DEVELOPMENT 60 FIGURE 6 THERAPEUTIC AREAS FOR PEDIATRIC DRUG APPROVALS SINCE THE IMPLEMENTATION OF PEDIATRIC REGULATIONS 60

4 LEADING THERAPEUTIC TARGETS 61 ALLERGY AND RESPIRATORY CONDITIONS 61 FIGURE 7 APPROVED ALLERGY AND RESPIRATORY TREATMENTS 61 TABLE 22 PEDIATRIC DRUGS APPROVED FOR RESPIRATORY CONDITIONS THROUGH PEDIATRIC REGULATORY PATHWAYS, 2013 ($ MILLIONS) TABLE 23 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR RESPIRATORY CONDITIONS, 2013 ($ MILLIONS/%) TABLE 24 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR RESPIRATORY CONDITIONS BY REGION, 2013 ($ MILLIONS) TABLE 25 GLOBAL SALES OF TOP FIVE PEDIATRIC DRUGS APPROVED FOR RESPIRATORY CONDITIONS, 2013 ($ MILLIONS/%) ANTI-INFECTIVES 65 FIGURE 8 APPROVED PEDIATRIC ANTI-INFECTIVES 66 TABLE 26 PEDIATRIC DRUGS APPROVED FOR INFECTIOUS DISEASES THROUGH PEDIATRIC REGULATORY PATHWAYS, 2013 ($ MILLIONS) TABLE 27 GLOBAL SALES OF ANTI-INFECTIVE PEDIATRIC DRUGS, 2013 ($ MILLIONS) 68 TABLE 28 GLOBAL SALES OF ANTI-INFECTIVE PEDIATRIC DRUGS APPROVED BY REGION, 2013 ($ MILLIONS) TABLE 29 GLOBAL SALES OF TOP FIVE PEDIATRIC DRUGS APPROVED FOR INFECTIOUS DISEASES, 2013 ($ BILLIONS/%) CENTRAL NERVOUS SYSTEM 69 FIGURE 9 APPROVED PEDIATRIC CNS MEDICATIONS 70 TABLE 30 PEDIATRIC DRUGS APPROVED FOR THE TREATMENT OF CNS CONDITIONS, 2013 ($ MILLIONS) TABLE 31 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR CNS CONDITIONS, 2013 ($ MILLIONS) TABLE 32 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR CNS CONDITIONS BY REGION, 2013 ($ MILLIONS) TABLE 33 GLOBAL SALES OF TOP FIVE PEDIATRIC DRUGS APPROVED FOR THE TREATMENT OF CNS CONDITIONS, 2013 ($ MILLIONS/%) PEDIATRIC VACCINES 73 FIGURE 10 APPROVED PEDIATRIC VACCINES 74 TABLE 34 LEADING PEDIATRIC AND ADOLESCENT VACCINES WORLDWIDE, 2013 ($ MILLIONS) TABLE 35 GLOBAL SALES OF VACCINES BY AGE/INDICATION, 2013 ($ MILLIONS) 76 TABLE 36 GLOBAL SALES OF PEDIATRIC VACCINES BY REGION, 2013 ($ MILLION) 76 TABLE 37 GLOBAL SALES OF TOP FIVE PEDIATRIC/ADOLESCENT VACCINES, 2013 ($ MILLIONS/%) ANTIDIABETIC THERAPIES 77 FIGURE 11 APPROVED PEDIATRIC ANTIDIABETIC THERAPIES 78 TABLE 38 LEADING PEDIATRIC ANTIDIABETIC THERAPIES, 2013 ($ MILLIONS) 79 TABLE 39 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR THE TREATMENT OF DIABETES BY ROUTE, 2013 ($ MILLIONS/%) TABLE 40 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR THE TREATMENT OF DIABETES BY REGION, 2013 ($ MILLIONS) TABLE 41 GLOBAL SALES OF TOP FIVE PEDIATRIC ANTIDIABETIC DRUGS, 2013 ($ MILLIONS/%) ORPHAN DRUG DISEASES 81 TABLE 42 APPROVED PEDIATRIC ORPHAN DRUGS IN THE U.S., ($) 82 FIGURE 12 ORPHAN DRUG DESIGNATIONS RECEIVING FDA MARKETING APPROVAL, (NUMBER)

5 TABLE 43 PEDIATRIC (CATEGORY 1) DISEASES WITH MOST DESIGNATIONS (NUMBER) FIGURE 13 ORPHAN PEDIATRIC PRODUCT APPROVALS, BY THERAPEUTIC CATEGORY (%) FIGURE 14 ORPHAN PEDIATRIC PRODUCT APPROVALS BY DRUG TYPE, (%) TABLE 44 PEDIATRIC ORPHAN DRUGS IN CLINICAL DEVELOPMENT BY ORPHAN DISEASE, COMPANY, PRODUCT AND PHASE TABLE 45 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED IN ORPHAN CONDITIONS BY THERAPY AREA, 2013 ($ MILLIONS) TABLE 46 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED IN ORPHAN CONDITIONS BY REGION, 2013 ($ MILLIONS/%) TABLE 47 GLOBAL SALES OF TOP FIVE PEDIATRIC DRUGS APPROVED IN ORPHAN CONDITIONS, 2013 ($ MILLIONS) PEDIATRIC ONCOLOGY 88 TABLE 48 GLOBAL APPROVED PEDIATRIC ONCOLOGY AGENTS IN EUROPE TABLE 49 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED IN ONCOLOGY CONDITIONS, BY REGION, 2013 ($ MILLIONS) TABLE 50 GLOBAL SALES OF TOP FIVE PEDIATRIC ONCOLOGY DRUGS, 2013 ($ MILLIONS) OTHER PEDIATRIC THERAPIES 91 TABLE 51 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR OTHER CONDITIONS, BY REGION, 2013 ($ MILLIONS/%) FIGURE 15 OTHER THERAPEUTIC AREAS OF PEDIATRIC PRODUCT APPROVAL CHAPTER 7 FUTURE OUTLOOK 94 MARKET TRENDS AND ANALYSIS 94 TABLE 52 GLOBAL SALES OF PEDIATRIC DRUGS BY THERAPY AREA, THROUGH 2019 ($ MILLIONS) REGIONAL TRENDS AND ANALYSIS 96 THE U.S. 96 FIGURE 16 ESTIMATED U.S. PEDIATRIC PRODUCT SALES MARKET SHARES, 2019 (%) 96 TABLE 53 U.S. SALES OF PEDIATRIC DRUGS BY THERAPEUTIC AREA, THROUGH 2019 ($ MILLIONS) EUROPE 97 FIGURE 17 ESTIMATED EUROPEAN PEDIATRIC PRODUCT MARKET SHARE, 2019 (%) 97 TABLE 54 EUROPEAN SALES OF PEDIATRIC DRUGS BY THERAPEUTIC AREA, JAPAN 98 FIGURE 18 ESTIMATED JAPANESE PEDIATRIC PRODUCT MARKET SHARE, 2019 (%) 99 TABLE 55 JAPANESE SALES OF PEDIATRIC DRUGS BY THERAPEUTIC AREA, REST OF WORLD (ROW) 100 FIGURE 19 ESTIMATED REST OF WORLD PEDIATRIC PRODUCT MARKET SHARES, 2019 (%) TABLE 56 REST OF WORLD SALES OF PEDIATRIC DRUGS BY THERAPEUTIC AREA, OVERALL CONCLUSIONS CHAPTER 8 COMPANY PROFILES 104

6 LEADING PEDIATRIC COMPANIES 104 PIERRE FABRE DERMATOLOGIE 104 SWEDISH ORPHAN BIOVITRUM (SOBI) 104 VIROPHARMA 105 LEADING CLINICAL RESEARCH ORGANIZATIONS 106 AGILITY CLINICAL, INC. 106 CLINIGENE INTERNATIONAL LIMITED 106 CONET (CLINICAL OPERATIONS NETWORK) 107 COVANCE INC. 107 INC RESEARCH 108 PAREXEL INTERNATIONAL CORP. 108 PPD 109 PREMIER RESEARCH 109 PSI CRO AG 110 QUINTILES 110 WWCT GLOBAL 111 LEADING PEDIATRIC NETWORKS 112 INTERNATIONAL NETWORKS 112 European Network of Paediatric Research (Enpr-EMA) 112 Global Research in Pediatrics (GRiP) 112 Programme for Global Paediatric Research (PGPR) 112 NATIONAL NETWORKS 113 Medicines for Clinical Research Network (MCRN) - United Kingdom 113 Medicines for Clinical Research Network (MCRN) - Netherlands 113 Finnish Investigators Network for Paediatric Medicines (FINPEDMED) - Finland Pediatric Trials Network (PTN) - United States CHAPTER 9 REFERENCES 116 CHAPTER 10 ACRONYMS 127

7 LIST OF TABLES TABLE HEADING SUMMARY TABLE GLOBAL PEDIATRIC MARKET, 7 TABLE 1 GLOBAL PEDIATRIC MARKET BY REGION, 10 TABLE 2 DISTRIBUTION OF WORLD POPULATION IN THE 0-14-YEAR AND YEAR AGE GROUPS BY REGION, 2013 AND 2050 (%) TABLE 3 DISTRIBUTION OF WORLD POPULATION BY REGION IN THE 0-14-YEAR AGE GROUP, (%) TABLE 4 DISTRIBUTION OF WORLD POPULATION IN THE 0-14-YEAR AGE GROUP, (MILLIONS) TABLE 5 OFF-LABEL CATEGORIES 13 TABLE 6 TYPES OF STUDIES REQUIRED WHEN MAKING A PIP SUBMISSION (NUMBER/%) TABLE 7 KEY AREAS OF UNMET PEDIATRIC NEED CITED BY THE EMA TABLE 8 DIFFERENCES BETWEEN ADULT AND PEDIATRIC TRIAL DESIGN 19 TABLE 9 AIMS OF THE PEDIATRIC FORMULATIONS INITIATIVE 20 TABLE 10 PERCENTAGE OF POPULATION BETWEEN 0 AND 14 YEARS OF AGE BY REGION, 2013 (%) TABLE 11 COMMON PEDIATRIC DISORDERS IN THE U.S. IN 2010 (MILLION/%) 24 TABLE 12 COMPARISON OF THE MAIN FEATURES OF THE U.S. PEDIATRIC INCENTIVE PROGRAMS TABLE 13 CLASSIFICATION OF PEDIATRIC AGE CATEGORIES IN THE EU AND THE U.S. 32 TABLE 14 PROCEDURAL GUIDELINES FOR PEDIATRIC DRUG DEVELOPMENT FOR EUROPE AND THE U.S. TABLE 15 SCIENTIFIC GUIDELINES FOR PEDIATRIC DRUG DEVELOPMENT FOR EUROPE AND THE U.S. TABLE 16 CURRENT LEGISLATIVE / REGULATORY PEDIATRIC INITIATIVES ACROSS KEY COUNTRIES GLOBALLY TABLE 17 KEY GLOBAL PEDIATRIC INITIATIVES 41 TABLE 18 PRIORITY MEDICINES FOR CHILDREN UNDER FIVE YEARS OF AGE FOR MAJOR CAUSES OF MORTALITY, MORBIDITY, PALLIATIVE CARE AND CHILD SURVIVAL TABLE 19 PRIORITY MEDICINES FOR CHILD HEALTH AND SURVIVAL IN NEED OF FURTHER RESEARCH AND DEVELOPMENT TABLE 20 TOP 10 COUNTRIES RANKED BY STUDY PARTICIPATION AND PATIENT ENROLLMENT FOR STUDIES ON DRUGS (NUMBER) TABLE 21 TOP 10 COUNTRIES RANKED BY STUDY PARTICIPATION AND PATIENT ENROLLMENT FOR STUDIES ON VACCINES (NUMBER) TABLE 22 PEDIATRIC DRUGS APPROVED FOR RESPIRATORY CONDITIONS THROUGH PEDIATRIC REGULATORY PATHWAYS, 2013 ($ MILLIONS) TABLE 23 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR RESPIRATORY CONDITIONS, 2013 ($ MILLIONS/%) TABLE 24 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR RESPIRATORY CONDITIONS BY REGION, 2013 ($ MILLIONS) TABLE 25 GLOBAL SALES OF TOP FIVE PEDIATRIC DRUGS APPROVED FOR RESPIRATORY CONDITIONS, 2013 ($ MILLIONS/%) TABLE 26 PEDIATRIC DRUGS APPROVED FOR INFECTIOUS DISEASES THROUGH PEDIATRIC REGULATORY PATHWAYS, 2013 ($ MILLIONS) TABLE 27 GLOBAL SALES OF ANTI-INFECTIVE PEDIATRIC DRUGS, 2013 ($ MILLIONS) 68 TABLE 28 GLOBAL SALES OF ANTI-INFECTIVE PEDIATRIC DRUGS APPROVED BY REGION, 2013 ($ MILLIONS) TABLE 29 GLOBAL SALES OF TOP FIVE PEDIATRIC DRUGS APPROVED FOR INFECTIOUS DISEASES, 2013 ($ BILLIONS/%)

8 TABLE HEADING TABLE 30 PEDIATRIC DRUGS APPROVED FOR THE TREATMENT OF CNS CONDITIONS, 2013 ($ MILLIONS) TABLE 31 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR CNS CONDITIONS, 2013 ($ MILLIONS) TABLE 32 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR CNS CONDITIONS BY REGION, 2013 ($ MILLIONS) TABLE 33 GLOBAL SALES OF TOP FIVE PEDIATRIC DRUGS APPROVED FOR THE TREATMENT OF CNS CONDITIONS, 2013 ($ MILLIONS/%) TABLE 34 LEADING PEDIATRIC AND ADOLESCENT VACCINES WORLDWIDE, 2013 ($ MILLIONS) TABLE 35 GLOBAL SALES OF VACCINES BY AGE/INDICATION, 2013 ($ MILLIONS) 76 TABLE 36 GLOBAL SALES OF PEDIATRIC VACCINES BY REGION, 2013 ($ MILLION) 76 TABLE 37 GLOBAL SALES OF TOP FIVE PEDIATRIC/ADOLESCENT VACCINES, 2013 ($ MILLIONS/%) TABLE 38 LEADING PEDIATRIC ANTIDIABETIC THERAPIES, 2013 ($ MILLIONS) 79 TABLE 39 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR THE TREATMENT OF DIABETES BY ROUTE, 2013 ($ MILLIONS/%) TABLE 40 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR THE TREATMENT OF DIABETES BY REGION, 2013 ($ MILLIONS) TABLE 41 GLOBAL SALES OF TOP FIVE PEDIATRIC ANTIDIABETIC DRUGS, 2013 ($ MILLIONS/%) TABLE 42 APPROVED PEDIATRIC ORPHAN DRUGS IN THE U.S., ($) 82 TABLE 43 PEDIATRIC (CATEGORY 1) DISEASES WITH MOST DESIGNATIONS (NUMBER) 84 TABLE 44 PEDIATRIC ORPHAN DRUGS IN CLINICAL DEVELOPMENT BY ORPHAN DISEASE, COMPANY, PRODUCT AND PHASE TABLE 45 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED IN ORPHAN CONDITIONS BY THERAPY AREA, 2013 ($ MILLIONS) TABLE 46 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED IN ORPHAN CONDITIONS BY REGION, 2013 ($ MILLIONS/%) TABLE 47 GLOBAL SALES OF TOP FIVE PEDIATRIC DRUGS APPROVED IN ORPHAN CONDITIONS, 2013 ($ MILLIONS) TABLE 48 GLOBAL APPROVED PEDIATRIC ONCOLOGY AGENTS IN EUROPE TABLE 49 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED IN ONCOLOGY CONDITIONS, BY REGION, 2013 ($ MILLIONS) TABLE 50 GLOBAL SALES OF TOP FIVE PEDIATRIC ONCOLOGY DRUGS, 2013 ($ MILLIONS) TABLE 51 GLOBAL SALES OF PEDIATRIC DRUGS APPROVED FOR OTHER CONDITIONS, BY REGION, 2013 ($ MILLIONS/%) TABLE 52 GLOBAL SALES OF PEDIATRIC DRUGS BY THERAPY AREA, THROUGH 2019 ($ MILLIONS) TABLE 53 U.S. SALES OF PEDIATRIC DRUGS BY THERAPEUTIC AREA, THROUGH 2019 ($ MILLIONS) TABLE 54 EUROPEAN SALES OF PEDIATRIC DRUGS BY THERAPEUTIC AREA, TABLE 55 JAPANESE SALES OF PEDIATRIC DRUGS BY THERAPEUTIC AREA, THROUGH 2019 ($ MILLIONS) TABLE 56 REST OF WORLD SALES OF PEDIATRIC DRUGS BY THERAPEUTIC AREA,

9 LIST OF FIGURES FIGURE TITLE SUMMARY FIGURE GLOBAL PEDIATRIC MARKET, ($ MILLIONS) 7 FIGURE 1 HISTORIC ANNUAL GROWTH RATES OF PEDIATRIC THERAPEUTIC SEGMENTS, (%) FIGURE 2 MAJOR MILESTONES OF PEDIATRIC LEGISLATION IN THE U.S FIGURE 3 GENERAL PRINCIPLES GUIDING PEDIATRIC DRUG DEVELOPMENT - THE ICH E-11 GUIDELINE FIGURE 4 MAJOR MILESTONES OF PEDIATRIC LEGISLATION IN THE EU FIGURE 5 PEDIATRIC AND ADULT TRIALS AS A PERCENTAGE, (%) 47 FIGURE 6 THERAPEUTIC AREAS FOR PEDIATRIC DRUG APPROVALS SINCE THE IMPLEMENTATION OF PEDIATRIC REGULATIONS FIGURE 7 APPROVED ALLERGY AND RESPIRATORY TREATMENTS 61 FIGURE 8 APPROVED PEDIATRIC ANTI-INFECTIVES 66 FIGURE 9 APPROVED PEDIATRIC CNS MEDICATIONS 70 FIGURE 10 APPROVED PEDIATRIC VACCINES 74 FIGURE 11 APPROVED PEDIATRIC ANTIDIABETIC THERAPIES 78 FIGURE 12 ORPHAN DRUG DESIGNATIONS RECEIVING FDA MARKETING APPROVAL, (NUMBER) FIGURE 13 ORPHAN PEDIATRIC PRODUCT APPROVALS, BY THERAPEUTIC CATEGORY (%) FIGURE 14 ORPHAN PEDIATRIC PRODUCT APPROVALS BY DRUG TYPE, (%) 85 FIGURE 15 OTHER THERAPEUTIC AREAS OF PEDIATRIC PRODUCT APPROVAL 91 FIGURE 16 ESTIMATED U.S. PEDIATRIC PRODUCT SALES MARKET SHARES, 2019 (%) 96 FIGURE 17 ESTIMATED EUROPEAN PEDIATRIC PRODUCT MARKET SHARE, 2019 (%) 97 FIGURE 18 ESTIMATED JAPANESE PEDIATRIC PRODUCT MARKET SHARE, 2019 (%) 99 FIGURE 19 ESTIMATED REST OF WORLD PEDIATRIC PRODUCT MARKET SHARES, 2019 (%)

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